Ruxolitinib-enhanced haploidentical transplant for children and young adults with sickle cell disease
Official title: Ruxolitinib-Enhanced Conditioning for Pediatric and Young Adult Patients With Symptomatic Sickle Cell Disease Undergoing Haploidentical Hematopoietic Cell Transplantation
Run by Arkansas Children's Hospital Research Institute · NCT07252050 · Data updated 2026-11-12
This trial will test whether adding ruxolitinib to a reduced-intensity haploidentical transplant lowers the risk of graft failure in children and young adults with sickle cell disease.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 12 Years to 45 Years |
| Sex | All |
| Enrollment | 24 (estimated) |
| Sponsor | Arkansas Children's Hospital Research Institute Academic / other |
| Drugs / interventions | ruxolitinib, cyclophosphamide, fludarabine |
| Locations | 4 sites (Aurora, Colorado and 3 other locations) |
| Status | Recruiting |
| Started | June 8, 2026 |
| Main results expected | November 19, 2028 |
| Study ends | November 19, 2029 |
| Registry record updated | June 4, 2026 |
| Trial ID | NCT07252050 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are patients aged 12–45 with any genotype of sickle cell disease who meet high-risk clinical criteria (for example stroke, recurrent acute chest syndrome, recurrent severe pain episodes, or need for chronic transfusions), have an HLA-haploidentical first-degree relative donor, and meet institutional HCT eligibility.
✕Probably not a fit
Patients who have an available HLA-matched sibling donor, are outside the 12–45 age range, or have contraindications to hematopoietic cell transplant or ruxolitinib are unlikely to benefit from this protocol.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
RUX-HAPLO is a Phase 1/2, single-arm, open-label multi-center trial enrolling up to 24 participants aged 12–45 undergoing haploidentical hematopoietic cell transplant for sickle cell disease. Participants receive cytoreduction with hydroxyurea followed by a reduced-intensity conditioning regimen (cyclophosphamide, fludarabine, thiotepa, ATG and low-dose TBI) and ruxolitinib started during conditioning and continued after transplant. Graft-versus-host disease prophylaxis includes post-transplant cyclophosphamide plus sirolimus or a calcineurin inhibitor. The primary endpoint is 1-year event-free survival with primary or secondary graft failure or death counted as events, and participants are followed for approximately two years post-transplant.
Why it matters
Potential benefit: If successful, this approach could reduce graft failure after haploidentical transplant and increase the chance of a durable cure for pediatric and young adult patients with sickle cell disease.
How similar studies have gone: Haploidentical reduced-intensity transplants have cured many patients with sickle cell disease and JAK inhibition with ruxolitinib has shown benefit for post-transplant inflammatory complications, but using ruxolitinib specifically to reduce graft failure in pediatric SCD is a relatively novel approach with limited prior data.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Participants with any genotypic form of SCD aged 12 - 45 years at enrollment with ≥1 of the following: 1. History of stroke and/or vasculopathy, including evidence of asymptomatic cerebrovascular disease for pediatric patients. 2. Recurrent moderate-severe acute chest syndrome (ACS) 3. Recurrent vaso-occlusive pain episodes requiring parenteral analgesia despite the institution of supportive care. 4. Need for chronic transfusion therapy to prevent vaso-occlusive complications (i.e. pain, stroke, and ACS). 5. For adult patients, an echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) ≥ 2.7 m/sec. 2. Participants must have an HLA haploidentical first degree relative (parent, sibling, or half sibling) who is willing and able to donate bone marrow. 3. Participants must meet institutional eligibility criteria for HCT. Exclusion Criteria: 1. Presence of an HLA-matched sibling who is willing and able to donate bone marrow. 2. Uncontrolled infection, evidence of active TB, Hepatitis B or C infection, or HIV seropositivity or infection. 3. Previous HCT or solid organ transplant. 4. CNS revascularization procedure, myocardial infarction, pulmonary embolus or deep vein thrombosis in the past 6 months. 5. Use of medications which significantly interfere with ruxolitinib metabolism. 6. Known hypersensitivity or severe reaction to ruxolitinib or any component of the conditioning regimen or its excipients. 7. Inability to swallow and retain oral medication (use of nasogastric or gastrostomy tube permitted). 8. History of malignancy except resected basal cell carcinoma or treated carcinoma in-situ. 9. Participation in another clinical trial involving an investigational or off-label use of a drug or device in the past 3 months. 10. Currently pregnant or breast feeding. 11. Clinically significant, uncontrolled autoimmune disease. 12. High-titer anti-donor specific HLA antibodies (without review and approval by Study Chair). 13. Participant (or guardian) inability or unwillingness to comply with the dose schedule and study evaluations, comprehend or sign informed consent and utilize a highly effective method of contraception (for participants of child-bearing potential). 14. Any condition that would, in the investigator's judgment, interfere with full participation in the study, pose a significant risk to the subject, or interfere with interpretation of study data.
Where it's running
-
Children's Healthcare of Atlanta
Atlanta, Georgia, United States Recruiting -
Children's Hospital of Colorado
Aurora, Colorado, United States Not yet recruiting -
Manning Family Children's
New Orleans, Louisiana, United States Not yet recruiting -
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States Not yet recruiting
Study contacts
- Principal investigator: Laura McLaughlin, MD — Arkansas Children's Hospital Research Institute
- Study coordinator: Laura McLaughlin, MD
- Email: LMcLaughlin@uams.edu
- Phone: 501-364-3908
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.